The National Organization for Rare Disorders is home base for more than 300 member organizations that serve not just patients, but providers who care for those with rare disorders, such as SMA and Duchenne muscular dystrophy.
Rare neurological disorders may have small prevalence numbers, but some patients affected by these conditions have an army of advocacy partners standing behind them. Sika Dunyoh, director of education programs at the National Organization for Rare Disorders (NORD), is one of them. In an interview with NeurologyLive, Dunyoh spoke about the active role of advocacy groups in diseases such as spinal muscular atrophy (SMA) and Duchenne muscular dystrophy, and how education—not just for patients, but providers–is key to improving outcomes in these populations.
Sika Dunyoh: We educate and support rare disease communities; We also advocate on their behalf on the Hill to protect their rights and to ensure that there are policies in place to encourage research and safe approval of therapies. We basically have the backs of rare disease patients and their families.
NORD is an umbrella organization, and we have nearly 300 disease-specific member organizations that serve the communities of the particular disease states. We have member organizations that focus specifically on SMA and Duchenne [muscular dystrophy], and these organizations are very active with regard to developing programs of support and advocacy for their patients. Like NORD, they're also very active and vocal on the Hill; they are pretty sophisticated operations and they're doing amazing work to serve their communities. There are a lot of disease states that don't have a patient organization and SMA and Duchenne are 2 disease states that are very blessed to have engaged patient organizations serving them.
For many years, a lot of our educational resources were designed for patients and caregivers, including our rare disease reports, our annual NORD summit, and occasional videos that we produce. In the last few years, we've focused more on educating providers. We had our first CME program a few years ago, and now we are expanding our CME offerings, so that's an area where we feel that we should be doing more to serve our population.
I think there is a spectrum of educational needs across the provider community; everything from information on rare diseases in general to topics around diagnosis so that we can see if we can shorten the path to diagnosis for patients. There are also educational needs that are very disease-specific like with SMA, Duchenne, Huntington disease, and other diseases where there needs to be education that can quicken diagnosis and improve clinical care. Across the board, we're trying to identify what those needs are and develop CME programming that will help move the needle on some of these issues.
We have other educational resources. For instance, we have a webinar series, and in it, we cover topics that cut across all rare diseases. In December, we had a webinar that gave tips on how to navigate insurance, reverse denials, and go through an appeal process with your insurance company. Those are the type of topics that help empower our patients so that they don't feel like the system is working against them. If they have tools and they have knowledge to be able to advocate on their own behalf, then they can see positive changes. We had a lot of great feedback on that webinar from people who said ‘I didn't even know I could do this!’ That's the kind of feedback and results that I want to see from our educational programs.
What I want them to know is that we are a resource for them. I think that a lot of providers don't consider the patient advocacy organizations as a potential resource, but we are. Through expanding our programming at NORD to include more CME, I think that we're going to be an even more useful resource for physicians and other medical professionals. I think that the ability to learn from us and to work with us will allow them to understand what the patient’s needs are and learn how to be better advocates on behalf of their patients.